The Pulse

OrphanPulse Wk 29: Ionis Takes SMA-Proven Antisense Into Dravet Syndrome

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OrphanPulse Wk 29 Ionis Takes SMA-Proven Antisense Into Dravet Syndrome

A week defined by first-in-human starts across rare genetic disease, led by antisense entering Dravet syndrome from the field that already rewrote spinal muscular atrophy. Around it, first patients dosed in rare vascular and cardiomyopathy programs with near-empty competitive fields, a first-in-class readout in heparin-induced thrombocytopenia, and orphan designations that quietly reshape the economics behind them.

NeuroPulse Wk 29: First Brain Link Shares Movement Between Two People

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NeuroPulse Wk 29 First Brain Link Shares Movement Between Two People

The Alzheimer’s Association International Conference drove the week’s pipeline news, but the signal that mattered most came from a lab: the first brain link that let one paralysed person move and feel through another. Around it, a tau target validated and contested in the same week, an oral entrant scaling in Huntington’s, and anti-amyloid therapy fighting its real battle, which is access.